News from Friday, February 28, 2025
Articles
Health Economics and Outcomes Research: Biopharma’s Underappreciated Value Creator
(2/28, Rob Abbott, MedCity News) comments “...HEOR needs to show that it cares about – and can meet – the needs of the biopharmaceutical company. I can’t escape the feeling that the recent layoffs took place, in part, because HEOR is viewed as a ‘nice to have’ not a ‘must have.’ When was the last time HEOR champions briefed company leadership on how fast evidence could be generated, or how data science and applied modeling techniques could accelerate access – and positive decisions – with HTA bodies, formulary decision makers, and regulators?” Full
Trump Blog: CMS Reiterates Drug Negotiation Guidance Ahead of Participation Deadline
(2/28, Nicole DeFeudis, Endpoints News) reports “...The agency published an infographic explaining the drug selection process, including how current guidance allows it to group together different products with the same active molecule (like Ozempic and Wegovy). Some legal and policy experts say the announcement could signal how the Trump administration intends to carry out negotiations going forward. The second round of negotiations is scheduled to kick off next week...Selected drugmakers have until Friday to decide if they are participating in the second round of negotiations.” Full
Press Releases
New Drug Approved for Inherited Clotting Disorder, Offering Simpler Dosing and Enhanced Quality of Life
(2/28, NICE Press Release) “NICE publishes final draft guidance recommending efanesoctocog alfa as an option for treating and preventing bleeding in people aged 2 and over with severe haemophilia A. Efanesoctocog alfa (also called Altuvoct and made by Swedish Orphan Biovitrum) can be given as a once-weekly injection instead of some current treatments that are given several times a week.” Full
Canada’s Drug Agency Publishes New Guidance for Rare Disease Registries in Canada
(2/28, Canada’s Drug Agency Press Release) “On Rare Disease Day — a global event dedicated to raising awareness for the millions of people living with rare diseases — Canada’s Drug Agency has published new pan-Canadian guidance on best practices and standards for rare disease registries (RDRs). The guidance, aligned with international recommendations, supports RDR holders to enhance the transparency, content, and quality of their data, and strengthen their capacity to contribute to health care decision-making.” Full
Journals
Centering Equity in Evidence-Informed Decision Making: Theoretical and Practical Considerations
Bomi Kim Hirsch, et al.
February 27, 2025, The Milbank Quarterly
The EU Project Real4Reg: Unlocking Real-World Data with AI
Jonas Peltner, et al.
February 27, 2025, Health Research Policy and Systems
Application of a Value Framework to Determine the Value of Prophylaxis Versus On-Demand Treatment in Adults with Hemophilia A in China
Baoying Tan, et al.
February 28, 2025, Advances in Therapy
Identifying Research Priorities for Localized, Recurrent, and Advanced Prostate Cancer Among Black Americans: A BACPAC Virtual Research Community Survey Analysis
Maheetha C Bharadwaj, et al.
March 2025, Urologic Oncology: Seminars and Original Investigations